NIMK — “Not In My Kid”
Did you know that many medicines are not approved by the FDA for use in children?
The FDA approves drugs for use based on scientific data from a series of studies. The type of study done to show whether a drug actually works or not is called a randomized clinical trial (RCT). In RCTs, people with a disease are randomized to either receive the study drug or not receive the study drug. The two groups are compared to see if the drug helped, hurt, or did nothing. (It’s way more complicated than that in real life but that’s the basic point.) The key to an RCT is that all patients in the study have an equal chance to receive the drug regardless of any characterisitics (age, gender, money, time they’ve been sick, etc.).
Once a drug is approved, the pharmaceutical company can market the drug for that application. So, for example, a drug that prevents seizures in people with epilepsy can be marketed as an anti-epileptic drug. Physicians can prescribe a drug for other uses. These are called “off-label” uses. Likewise, drugs can be marketed for use in the population in which they’ve been tested. So, a drug tested in adults can’t be marketed for use in children but doctors can use the drug in children if they feel it’s appropriate.
A study earlier this year found that 77% of parents only want drugs given to their children that have been approved by the FDA for use in children. Makes sense … better safe than sorry. However, only 30% of the same parents would allow their own children to be enrolled in an RCT!
The researchers concluded that parents didn’t understand the safeguards put in place to protect children in studies and that educating the parents might increase participation.
I don’t know. I’m an epidemiologist and I’ve participated in clinical trials from both sides (patient and researcher). I can understand the hesitation to put one’s child in a study. I’m willing to bet that participation is much higher for diseases in which there is no other option like rare, aggressive cancers. If there’s an adequate, available, well-studied treatment available, it would be difficult to risk your child’s health. I think I’d be more likely to participate if the study period was very short with a quick transfer to the usual treatment if the study treatment (drug or placebo) wasn’t working. And I’d probably do a lot of reading about the study treatment but then I have the access to basic science research and the medical literature plus the education to understand what I’m reading. An average parent would just have to trust the doctors/researchers.
Would you enroll your child in a clinical trial?
